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NCT03201965 · OUTCOME

Organ Response Rate (OrRR) at 6 Months: Cardiac Response

A Study to Evaluate the Efficacy and Safety of Daratumumab in Combination With Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) Compared to CyBorD Alone in Newly Diagnosed Systemic Amyloid Light-chain (AL) Amyloidosis · Source last updated 2025-11-26

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
NUMBER
Unit
Percentage of participants
Interval / dispersion
95% Confidence Interval
Time frame
Month 6

What was measured

The Organ Response Rate (OrRR) for heart was defined as the percentage of participants with baseline involvement of the heart who achieved a confirmed organ response in the heart. Cardiac response was defined as as a decrease of \>30% in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels and greater than (\>)300 nanogram/Litre (ng/L).

Analysis population: Cardiac response-evaluable participants had baseline NT-proBNP \>=650 ng/L or NYHA class 3 or 4, plus received at least 1 administration of study treatment and had at least one post-baseline. NT-proBNP measurement (if baseline NT-proBNP \>=650 nanograms per liter \[ng/L\]) or NYHA function evaluation (if baseline NYHA class 3 or 4). Data for this outcome measure was planned to be collected and analyzed for specified arms only.

Groups in this outcome

CyBorD

Participants received dexamethasone (40 mg weekly dose)starting from cycle 1 day1 in every 28-day cycle followed by cyclophosphamide 300 mg/m\^2 orally or IV maximum weekly dose 500 mg and then bortezomib 1.3 mg/m\^2 SC injection once weekly up to 7.33 months. All cycles were of 28 days. After completion of treatment phase participants entered into the post treatment observation phase and were continued with disease evaluations every 8 weeks post last dose up to disease progression (up to 6.5 years). Participants then entered into long term follow up phase and were followed up for survival every 16 weeks (up to 7.1 years).

Daratumumab Plus CyBorD

Participants received combination therapy for six cycles of 28 days each, daratumumab 1800 mg SC weekly starting from cycles 1 day 1 and cycle 2, every 2 weeks in cycles 3 through 6, dexamethasone 40 mg weekly (on days of daratumumab dosing, dexamethasone was split, 20 mg as premedication and 20 mg as post daratumumab dosing), cyclophosphamide 300 mg/m\^2 orally or IV maximum weekly dose 500 mg, bortezomib 1.3 mg/m\^2 SC injection once weekly. After 6 cycles, participants continued to receive daratumumab SC monotherapy until disease progression, start of subsequent therapy or up to 26.71 months from the first dose of study treatment. All cycles were of 28 days. After completion of treatment phase participants entered into the post treatment observation phase and were continued with disease evaluations every 8 weeks post last dose up to disease progression (up to 6.5 years). Participants then entered into long term follow up phase and were followed up for survival every 16 weeks (up to 7.1 years).

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
CyBorD117
Daratumumab Plus CyBorD118

Reported measurements

Source class 1
Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
CyBorD22.2Not reported15.130.8Not reported
Daratumumab Plus CyBorD41.5Not reported32.551.0Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        15.1
        upper Limit
        30.8
        value
        22.2
      2. group Id
        OG001
        lower Limit
        32.5
        upper Limit
        51.0
        value
        41.5
denoms
  1. counts
    1. group Id
      OG000
      value
      117
    2. group Id
      OG001
      value
      118
    units
    Participants
description
The Organ Response Rate (OrRR) for heart was defined as the percentage of participants with baseline involvement of the heart who achieved a confirmed organ response in the heart. Cardiac response was defined as as a decrease of \>30% in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels and greater than (\>)300 nanogram/Litre (ng/L).
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants received dexamethasone (40 mg weekly dose)starting from cycle 1 day1 in every 28-day cycle followed by cyclophosphamide 300 mg/m\^2 orally or IV maximum weekly dose 500 mg and then bortezomib 1.3 mg/m\^2 SC injection once weekly up to 7.33 months. All cycles were of 28 days. After completion of treatment phase participants entered into the post treatment observation phase and were continued with disease evaluations every 8 weeks post last dose up to disease progression (up to 6.5 years). Participants then entered into long term follow up phase and were followed up for survival every 16 weeks (up to 7.1 years).
    id
    OG000
    title
    CyBorD
  2. description
    Participants received combination therapy for six cycles of 28 days each, daratumumab 1800 mg SC weekly starting from cycles 1 day 1 and cycle 2, every 2 weeks in cycles 3 through 6, dexamethasone 40 mg weekly (on days of daratumumab dosing, dexamethasone was split, 20 mg as premedication and 20 mg as post daratumumab dosing), cyclophosphamide 300 mg/m\^2 orally or IV maximum weekly dose 500 mg, bortezomib 1.3 mg/m\^2 SC injection once weekly. After 6 cycles, participants continued to receive daratumumab SC monotherapy until disease progression, start of subsequent therapy or up to 26.71 months from the first dose of study treatment. All cycles were of 28 days. After completion of treatment phase participants entered into the post treatment observation phase and were continued with disease evaluations every 8 weeks post last dose up to disease progression (up to 6.5 years). Participants then entered into long term follow up phase and were followed up for survival every 16 weeks (up to 7.1 years).
    id
    OG001
    title
    Daratumumab Plus CyBorD
param Type
NUMBER
population Description
Cardiac response-evaluable participants had baseline NT-proBNP \>=650 ng/L or NYHA class 3 or 4, plus received at least 1 administration of study treatment and had at least one post-baseline. NT-proBNP measurement (if baseline NT-proBNP \>=650 nanograms per liter \[ng/L\]) or NYHA function evaluation (if baseline NYHA class 3 or 4). Data for this outcome measure was planned to be collected and analyzed for specified arms only.
reporting Status
POSTED
time Frame
Month 6
title
Organ Response Rate (OrRR) at 6 Months: Cardiac Response
type
SECONDARY
unit Of Measure
Percentage of participants

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